Amplifying the Body’s Neural Stem Cells May Help Treat Lou Gehrig’s Disease

Stem cells within the spinal cord and brain can do something once thought impossible: replace damaged nerve cells. A University of Washington research team is investigating whether the reparative action of neural stem cells can be enhanced sufficiently to treat amyotrophic lateral sclerosis (ALS), commonly referred to as Lou Gehrig’s disease.

“There’s good data showing that the human brain has a limited capacity to renew its own neurons by using stem cells already present in the nervous system,” said Dr. Michel Kliot, associate professor of neurological surgery. “We’re trying to amplify this response to the point where we can replace dying neurons affected by the disease.”

Lou Gehrig’s disease, named after the baseball player who died with the disorder in 1941, is a progressive neurological disease that kills motor neurons, the nerve cells that control muscle movement and power. It often strikes during mid-life with slowly advancing paralysis; patients usually die within five years.

The UW research expands on work at the University of California, Irvine. In an animal model of Parkinson’s disease, researchers there injected transforming growth factor alpha, a protein that stimulates cell growth, into the brain. The injection spurred stem cells to develop into neurons that make dopamine, the neurotransmitter absent in Parkinson’s patients.

Collaborating with the Irvine team, Kliot and his colleagues began parallel work in an animal model for Lou Gehrig’s disease. The hypothesis is that application of transforming growth factor alpha may enable stem cells to develop into replacement neurons in response to the injury signals from dying motor neurons.

“If amplification of the stem cell response works,” said Kliot, “It’s potentially applicable to a variety of other brain disorders.”

UW research on amyotrophic lateral sclerosis (ALS) has benefited this year from contributions from national ALS organizations. The Department of Neurological Surgery received gifts from Project A.L.S. to support stem cell research, and the Department of Neurology received contributions from the ALS Therapy Development Fund, a non-profit biotechnology company. Both contributors, located on the East Coast, recognized the UW’s reputation for excellence in research.